Eidos Therapeutics commenced a pivotal Phase III clinical trial of its investigational small molecule AG10 for the treatment of transthyretin (TTR) amyloid cardiomyopathy (ATTR-CM).
ATTR is caused by tetrameric transthyretin (TTR) destabilisation due to inherited mutations or aging. AG10 is designed to stabilise TTR and prevent the molecular events leading to amyloidosis (ATTR).
The global Phase III trial is dubbed ATTRibute-CM and will compare 800mg AG10 with placebo in a total of 510 patients suffering from symptomatic ATTR-CM associated with either wild-type or mutant TTR.
German biopharmaceutical company Immatics is set to study its investigational therapy IMA101 in combination with Roche’s Tecentriq (atezolizumab) drug for the treatment of solid cancers.
Based on Immatics’ ACTolog approach, IMA101 is a personalised, multi-targeted autologous cell therapy intended for various advanced / metastatic solid tumours.
Tecentriq is a monoclonal antibody that binds to the PD-L1 protein and prevents its interaction with PD-1 and B7.1 receptors. The inhibition of PD-L1 is expected to activate T cells.
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The US Food and Drug Administration (FDA) issued a warning regarding pulmonary embolism risk and increased overall mortality in Pfizer’s postmarketing trial assessing Xeljanz (tofacitinib) in rheumatoid arthritis patients.
The trial is intended to investigate the safety of 5mg and 10mg twice-daily Xeljanz, particularly the risk of cardiovascular events, cancer, and opportunistic infections.
A review of study data by the Data Safety Monitoring Board revealed the safety risks in patients administered with 10mg of the drug.
Swiss gene editing company CRISPR Therapeutics and US-based Vertex Pharmaceuticals started treating patients in a Phase I/II clinical trial of an investigational gene-editing therapy CTX001 for the treatment of transfusion-dependent beta thalassemia (TDT).
CTX001 is an autologous, CRISPR/Cas9 gene-edited hematopoietic stem cell therapy being developed to help patients suffering from severe hemoglobinopathies.
The product is the first therapy under a research collaboration formed by CRISPR and Vertex in 2015 to use CRISPR/Cas9 for developing new treatments targeting underlying genetic causes of disease.
Omecamtiv mecarbil is a selective cardiac myosin activator designed to bind to the catalytic domain of myosin. It is being co-developed by Amgen and Cytokinetics, with funding and support from Servier.
The randomised, parallel group, multi-centre METEORIC-HF trial will assess the effect of omecamtiv mecarbil on exercise capacity, compared to placebo.
Intercept Pharmaceuticals reported positive top-line results from the pivotal Phase III REGENERATE clinical trial of obeticholic acid (OCA) in patients with liver fibrosis caused due to non-alcoholic steatohepatitis (NASH).
Primary efficacy analysis showed fibrosis improvement with no worsening of NASH, when treated with once-daily 25mg OCA, at 18 months. The analysis was carried out in a total of 931 patients.
The trial met the primary endpoint of NASH resolution with no worsening of liver fibrosis in more patients in OCA arms, compared to placebo, but did not achieve statistical significance.
Novus Therapeutics commenced dosing in a Phase IIa clinical trial of OP0201 for the treatment of otitis media, an inflammatory disease affecting the middle ear.
OP0201 is a drug-device combination that consists of a formulation of surfactant dipalmitoylphosphatidylcholine and a spreading agent called cholesteryl palmitate, suspended in a propellant.
The drug is administered intranasally using a pressurised metered-dose inhaler (pMDI). It is meant to restore the normal physiologic activity of the Eustachian tube (ET), which connects the middle ear to the back of the nasopharynx.
CStone Pharmaceuticals is set to initiate a Phase III clinical trial to study its avapritinib drug candidate as a third-line treatment for KIT-driven gastrointestinal stromal tumours (GIST).
The Chinese company secured the National Medical Products Administration (NMPA) approval to conduct the trial in China.
Avapritinib is an oral, potent and highly selective KIT and PDGFRα inhibitor. Preclinical data demonstrated that the therapeutic is active against a broad spectrum of KIT and PDGFRα mutations.
US-based biopharmaceutical firm Denali Therapeutics started dosing patients with its investigational drug DNL747 in a Phase Ib clinical trial for the treatment of Alzheimer’s disease.
DNL747 is designed to target the receptor-interacting serine / threonine-protein kinase 1 (RIPK1) protein, which is associated with signalling in the TNF receptor pathway that controls inflammation and cell death in tissues.
Denali is developing various RIPK1-targeting molecules in collaboration with Sanofi, including DNL747 for Alzheimer’s disease, amyotrophic lateral sclerosis (ALS) and multiple sclerosis.
Ascendis Pharma submitted an investigational new drug (IND) application to the US Food and Drug Administration (FDA) seeking approval to conduct a Phase II clinical trial of TransCon PTH for the treatment of hypoparathyroidism (HP).
TransCon PTH is designed as a long-acting prodrug of parathyroid hormone (PTH[1-34]). It is intended to provide replacement therapy for HP.
The company’s TransCon technology is used to develop new therapies that optimise therapeutic effect. Molecules created using the platform comprise an unmodified parent drug, an inert carrier to protect it, and a linker that temporarily binds the drug and carrier.